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ZMYND11 相关综合征性智力障碍:16 例患者的表型谱描绘和扩展。

ZMYND11-related syndromic intellectual disability: 16 patients delineating and expanding the phenotypic spectrum.

机构信息

Sheffield Clinical Genetics Service, Sheffield Children's NHS Foundation Trust, Sheffield, UK.

Department of Pediatrics, Johns Hopkins University, Baltimore, Maryland.

出版信息

Hum Mutat. 2020 May;41(5):1042-1050. doi: 10.1002/humu.24001. Epub 2020 Mar 5.

Abstract

Pathogenic variants in ZMYND11, which acts as a transcriptional repressor, have been associated with intellectual disability, behavioral abnormalities, and seizures. Only 11 affected individuals have been reported to date, and the phenotype associated with pathogenic variants in this gene have not been fully defined. Here, we present 16 additional patients with predicted pathogenic heterozygous variants in including four individuals from the same family, to further delineate and expand the genotypic and phenotypic spectrum of ZMYND11-related syndromic intellectual disability. The associated phenotype includes developmental delay, particularly affecting speech, mild-moderate intellectual disability, significant behavioral abnormalities, seizures, and hypotonia. There are subtle shared dysmorphic features, including prominent eyelashes and eyebrows, a depressed nasal bridge with bulbous nasal tip, anteverted nares, thin vermilion of the upper lip, and wide mouth. Novel features include brachydactyly and tooth enamel hypoplasia. Most identified variants are likely to result in premature truncation and/or nonsense-mediated decay. Two ZMYND11 variants located in the final exon-p.(Gln586*) (likely escaping nonsense-mediated decay) and p.(Cys574Arg)-are predicted to disrupt the MYND-type zinc-finger motif and likely interfere with binding to its interaction partners. Hence, the homogeneous phenotype likely results from a common mechanism of loss-of-function.

摘要

ZMYND11 中的致病性变异可导致智力障碍、行为异常和癫痫,该基因作为转录抑制剂发挥作用。迄今为止,仅报道了 11 例受影响的个体,且该基因中的致病性变异相关表型尚未完全定义。在此,我们报告了另外 16 例预测为致病性杂合变异的患者,包括来自同一家庭的 4 名个体,以进一步描述和扩展 ZMYND11 相关综合征性智力障碍的基因型和表型谱。相关表型包括发育迟缓,特别是言语发育迟缓、轻度至中度智力障碍、严重的行为异常、癫痫和肌张力减退。存在一些细微的共同畸形特征,包括睫毛和眉毛浓密、鼻骨扁平、鼻尖呈球状、鼻孔前翻、上唇红唇薄、口型宽大。新发现的特征包括短指畸形和牙釉质发育不全。大多数鉴定出的变异可能导致过早截断和/或无义介导的衰变。两个位于最后一个外显子的 ZMYND11 变异体-p.(Gln586*)(可能逃避无义介导的衰变)和 p.(Cys574Arg)-预计会破坏 MYND 型锌指结构域,并可能干扰与相互作用伙伴的结合。因此,同质表型可能是由于共同的功能丧失机制所致。

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